Neuronata-R® ALS Phase 3 Data Supports FDA Accelerated Approval Bid

Neuronata-R® ALS Phase 3 Data Supports FDA Accelerated Approval Bid

By, Admin 4 June 2025

Seoul, South Korea – June 4, 2025 – CorestemChemon Inc. (KOSDAQ: 166480) today announced promising subgroup findings from its Phase 3 ALSummit trial (NCT04745299) of Neuronata-R® (Lenzumestrocel), an autologous bone marrow-derived mesenchymal stem cell (MSC) therapy for amyotrophic lateral sclerosis (ALS).

While the trial did not meet its primary endpoint across the overall study population, statistically significant efficacy signals emerged in a pre-defined subgroup of participants with slow disease progression, including improvements in function, survival, respiratory capacity, and reduction in neurofilament light chain (NfL) — a biomarker already used to support FDA accelerated approval in ALS.

Subgroup Efficacy: Functional and Respiratory Benefits in Early-Stage ALS

In the slow-progressing ALS subgroup, Neuronata-R® demonstrated significant improvements in:

  • CAFS (Combined Assessment of Function and Survival)
  • ALSFRS-R functional scores
  • Slow Vital Capacity (SVC), a key respiratory measure

Participants in the five-dose treatment group showed earlier and greater functional improvement than those in the two-dose group. At Month 6, statistically significant CAFS improvements were observed in both treatment arms versus placebo:

  • Group 1 (two doses): LS Mean 20.95 vs 13.66 (p < 0.024)
  • Group 2 (five doses): LS Mean 24.78 vs 17.92 (p < 0.041)

SVC improvements in Group 2 emerged by Month 8, indicating a potential delay in disease progression.

NfL Biomarker Reduction Strengthens Regulatory Pathway

Both treatment arms showed sustained reductions in NfL, a validated ALS biomarker. Notably, Group 2 demonstrated significant reductions at Month 4 and Month 10 versus placebo, suggesting a dose-dependent biological effect.

This finding mirrors the FDA’s recent accelerated approval of Tofersen, which was based on NfL reduction rather than direct survival benefit — a precedent CorestemChemon intends to leverage in its own accelerated approval strategy.

Regulatory Outlook: BLA Submission Targeted for Q4 2025

CorestemChemon plans to hold a Pre-BLA or Type C meeting with the FDA in Q3 2025 to discuss its regulatory strategy. The company aims to submit a Biologics License Application (BLA) by the end of 2025, with potential FDA accelerated approval targeted for mid-2026.

A company official stated:

“This subgroup analysis provides compelling evidence to support a biomarker-driven approval strategy. The alignment between our internal data and independent CRO validation strengthens our regulatory position.”

About Neuronata-R® (Lenzumestrocel)

Neuronata-R® is an autologous MSC therapy derived from the patient’s own bone marrow. The cells modulate neuroinflammation, protect motor neurons, and secrete therapeutic paracrine factors. To date, the therapy has been administered to over 400 commercial patients and 190 trial participants without serious treatment-related adverse events.

Neuronata-R® holds Orphan Drug Designation from both the U.S. FDA (2018) and the European Medicines Agency (2019). It received initial marketing approval from South Korea’s MFDS in 2014 and completed Phase 3 in 2024.

About CorestemChemon Inc.

Founded in 2002, CorestemChemon Inc. is a South Korea-based biotech company pioneering stem cell-based therapeutics for neurodegenerative diseases. The company is focused on developing innovative, patient-derived cell therapies to address unmet medical needs in ALS and related conditions.

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